U.S. Food and Drug Administration Approves KALYDECO (ivacaftor) for Use in Eight Additional Mutations that Cause Cystic Fibrosis

KALYDECO, the first drug approved to treat the underlying genetic defect that causes cystic fibrosis, was approved in January 2012 to treat patients ages 6 and older with one type of CFTR genetic mutation – G551D. The medication works to increase the opening of the CFTR protein to improve chloride transport and alleviate symptoms of CF.
Because the drug has been effective in these patients, efforts have been made to study its benefits in a larger population. A recent study of 39 patients with CF showed significant improvement in FEV1 for patients in the presence of additional genetic mutations.

On February 21, 2014, the results of this study led Vertex Pharmaceuticals to announce the FDA approval of KALYDECO for use in CF patients with any of 8 additional CFTR gene mutations. In the U.S., 150 persons >6 years of age have one of these new mutations and approximately 250 people in Europe and Australia do as well. The additional mutations included in the FDA-approved use of KALYDECO include:

  • G178R
  • S549R
  • S549N
  • G551S
  • G1244E
  • S1251N
  • S1255P
  • G1349D

Senior Vice President and Co-Chief Medical Officer at Vertex said, “We believe that KALYDECO has the potential to help more people with CF, and today’s approval is an important step toward that goal.” Currently, other studies are in progress to evaluate if KALYDECO can benefit even more patients with CF.